136 Participants Needed

AAT + Corticosteroids for Graft-versus-Host Disease

Recruiting at 25 trial locations
TR
Overseen ByTrial Registration Coordinator
Age: Any Age
Sex: Any
Trial Phase: Phase 3
Sponsor: CSL Behring
Must be taking: Corticosteroids
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

What is the purpose of this trial?

Study CSL964_5001 will investigate the efficacy of AAT with corticosteroids compared with corticosteroids alone as first line therapy for patients with high-risk acute GVHD

Research Team

SD

Study Director

Principal Investigator

CSL Behring

Eligibility Criteria

This trial is for patients aged 12 or older who have acute GVHD after a stem cell transplant and need systemic therapy with corticosteroids. It's not for those previously treated with AAT, having cancer that's returned or not responding to treatment, using steroids within the last week before GVHD onset, or currently on other drugs for GVHD.

Inclusion Criteria

I am 12 years old or older.
I have had a transplant from any donor or undergone any intensity of preparation treatment.
I have acute GVHD after a stem cell transplant.
See 1 more

Exclusion Criteria

My cancer has come back or is getting worse.
I have chronic GVHD or overlap syndrome that started before or at the time of joining.
I started taking systemic corticosteroids within 7 days before my acute GVHD symptoms began.
See 2 more

Treatment Details

Interventions

  • Alpha-1 antitrypsin (AAT)
  • Placebo
Trial OverviewThe study compares two treatments for acute GVHD: one group receives Alpha-1 antitrypsin (AAT) plus corticosteroids, while the other gets a placebo alongside corticosteroids. The goal is to see if adding AAT improves outcomes.
Participant Groups
2Treatment groups
Experimental Treatment
Placebo Group
Group I: AATExperimental Treatment1 Intervention
Alpha-1 antitrypsin (AAT) is a lyophilized powder for intravenous administration
Group II: PlaceboPlacebo Group1 Intervention
Albumin solution administered intravenously

Alpha-1 antitrypsin (AAT) is already approved in United States, European Union for the following indications:

🇺🇸
Approved in United States as Alpha-1 antitrypsin for:
  • Chronic obstructive pulmonary disease (COPD) associated with alpha-1 antitrypsin deficiency
🇪🇺
Approved in European Union as Alpha-1 antitrypsin for:
  • Chronic obstructive pulmonary disease (COPD) associated with alpha-1 antitrypsin deficiency

Find a Clinic Near You

Who Is Running the Clinical Trial?

CSL Behring

Lead Sponsor

Trials
204
Recruited
1,207,000+
Dr. Paul McKenzie profile image

Dr. Paul McKenzie

CSL Behring

Chief Executive Officer since 2023

PhD in Chemical Engineering from Carnegie Mellon University, B.S. in Chemical Engineering from the University of Pennsylvania

Dr. Bill Mezzanotte profile image

Dr. Bill Mezzanotte

CSL Behring

Chief Medical Officer since 2021

MD from Duke University

National Institutes of Health (NIH)

Collaborator

Trials
2,896
Recruited
8,053,000+

National Cancer Institute (NCI)

Collaborator

Trials
14,080
Recruited
41,180,000+

Blood and Marrow Transplant Clinical Trials Network

Collaborator

Trials
51
Recruited
14,600+

National Heart, Lung, and Blood Institute (NHLBI)

Collaborator

Trials
3,987
Recruited
47,860,000+